| NCT04042025 |
Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi |
https://ClinicalTrials.gov/show/NCT04042025 |
Enrolling by invitation |
AveXis, Inc. |
2034-12-31 |
| NCT03395795 |
Trial Evaluating the Interest of Noninvasive Ventilation in NAVA Mode in Respiratory Decompensations Children With Infantile Spinal Muscular Atrophy Type II |
https://ClinicalTrials.gov/show/NCT03395795 |
Recruiting |
Assistance Publique - Hôpitaux de Paris |
2020-02-29 |
| NCT03819660 |
Long Term Safety of Amifampridine Phosphate in Spinal Muscular Atrophy 3 |
https://ClinicalTrials.gov/show/NCT03819660 |
Enrolling by invitation |
Catalyst Pharmaceuticals, Inc. |
2020-12-31 |
| NCT03306277 |
Gene Replacement Therapy Clinical Trial for Patients With Spinal Muscular Atrophy Type 1 |
https://ClinicalTrials.gov/show/NCT03306277 |
Completed |
AveXis, Inc. |
2019-11-12 |
| NCT03223051 |
Development of a Space Exploration Assessment for Children With Spinal Muscular Atrophy |
https://ClinicalTrials.gov/show/NCT03223051 |
Completed |
Centre d’Investigation Clinique et Technologique 805 |
2019-11-22 |
| NCT03032172 |
A Study of Risdiplam (RO7034067) in Adult and Pediatric Participants With Spinal Muscular Atrophy |
https://ClinicalTrials.gov/show/NCT03032172 |
Active, not recruiting |
Hoffmann-La Roche |
2021-12-31 |
| NCT02941328 |
SPACE Trial: Pyridostigmine vs Placebo in SMA Types 2, 3 and 4 |
https://ClinicalTrials.gov/show/NCT02941328 |
Completed |
UMC Utrecht |
2018-01-31 |
| NCT02913482 |
Investigate Safety, Tolerability, PK, PD and Efficacy of Risdiplam (RO7034067) in Infants With Type1 Spinal Muscular Atrophy |
https://ClinicalTrials.gov/show/NCT02913482 |
Active, not recruiting |
Hoffmann-La Roche |
2019-11-14 |
| NCT02876094 |
Effect of Low-Dose Celecoxib on SMN2 in Patients With Spinal Muscular Atrophy |
https://ClinicalTrials.gov/show/NCT02876094 |
Recruiting |
Children’s Hospital of Eastern Ontario |
2020-07-31 |
| NCT02644668 |
A Study of CK-2127107 in Patients With Spinal Muscular Atrophy |
https://ClinicalTrials.gov/show/NCT02644668 |
Completed |
Cytokinetics |
2018-05-31 |
| NCT02633709 |
A Study to Investigate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Risdiplam (RO7034067) Given by Mouth in Healthy Volunteers |
https://ClinicalTrials.gov/show/NCT02633709 |
Completed |
Hoffmann-La Roche |
2016-08-04 |
| NCT02628743 |
A Study to Evaluate Long Term Safety, Tolerability, and Effectiveness of Olesoxime in Patients With Spinal Muscular Atrophy (SMA) |
https://ClinicalTrials.gov/show/NCT02628743 |
Completed |
Hoffmann-La Roche |
2018-12-18 |
| NCT02594124 |
A Study for Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in Nusinersen (ISIS 396443) Investigational Studies. |
https://ClinicalTrials.gov/show/NCT02594124 |
Active, not recruiting |
Biogen |
2023-08-29 |
| NCT04089566 |
Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy |
https://ClinicalTrials.gov/show/NCT04089566 |
Recruiting |
Biogen |
2022-09-26 |
| NCT02391831 |
Prospective Study of the Natural History of Patients With Type 2 and 3 Spinal Muscular Atrophy |
https://ClinicalTrials.gov/show/NCT02391831 |
Completed |
Institut de Myologie, France |
2018-06-30 |
| NCT02386553 |
A Study of Multiple Doses of Nusinersen (ISIS 396443) Delivered to Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy |
https://ClinicalTrials.gov/show/NCT02386553 |
Active, not recruiting |
Biogen |
2025-02-25 |
| NCT02341053 |
Effects of Standing on Non-Ambulatory Children With Spinal Muscular Atrophy |
https://ClinicalTrials.gov/show/NCT02341053 |
Active, not recruiting |
Gillette Children’s Specialty Healthcare |
2019-12-31 |
| NCT02292537 |
A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Participants With Later-onset Spinal Muscular Atrophy (SMA) |
https://ClinicalTrials.gov/show/NCT02292537 |
Completed |
Biogen |
2017-02-20 |
| NCT02268552 |
An Open Label Study of LMI070 (Branaplam) in Type 1 Spinal Muscular Atrophy (SMA) |
https://ClinicalTrials.gov/show/NCT02268552 |
Active, not recruiting |
Novartis |
2020-07-23 |
| NCT02122952 |
Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1 |
https://ClinicalTrials.gov/show/NCT02122952 |
Completed |
AveXis, Inc. |
2017-12-15 |
| NCT02061189 |
Pilot Study of an Innovative Physiotherapy in Patients With Infantile Spinal Muscular Atrophy (SMA) |
https://ClinicalTrials.gov/show/NCT02061189 |
Active, not recruiting |
Assistance Publique - Hôpitaux de Paris |
2020-04-30 |
| NCT01302600 |
Safety and Efficacy of Olesoxime (TRO19622) in 3-25 Years SMA Patients. |
https://ClinicalTrials.gov/show/NCT01302600 |
Completed |
Hoffmann-La Roche |
2013-10-31 |
| NCT02052791 |
An Open-label Safety and Tolerability Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in ISIS 396443-CS2 (NCT01703988) or ISIS 396443-CS10 (NCT01780246) |
https://ClinicalTrials.gov/show/NCT02052791 |
Completed |
Biogen |
2017-01-31 |
| NCT02003937 |
Aerobic Training in Patients With Spinal Muscular Atrophy Type III |
https://ClinicalTrials.gov/show/NCT02003937 |
Completed |
Rigshospitalet, Denmark |
2010-09-30 |
| NCT01981915 |
Optimum Insufflation Capacity in NMD |
https://ClinicalTrials.gov/show/NCT01981915 |
Completed |
Universität Duisburg-Essen |
2013-02-28 |
| NCT01862042 |
Palliative Care in Spinal Muscular Atrophy (SMA) 1 |
https://ClinicalTrials.gov/show/NCT01862042 |
Completed |
Assistance Publique - Hôpitaux de Paris |
2018-06-11 |
| NCT01839656 |
A Study to Assess the Efficacy, Safety and Pharmacokinetics of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy (SMA) |
https://ClinicalTrials.gov/show/NCT01839656 |
Completed |
Biogen |
2017-08-21 |
| NCT01780246 |
An Open-label Safety and Tolerability Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy Who Previously Participated in ISIS 396443-CS1 (NCT01494701) |
https://ClinicalTrials.gov/show/NCT01780246 |
Completed |
Biogen |
2014-02-28 |
| NCT01703988 |
An Open-label Safety, Tolerability and Dose-Range Finding Study of Multiple Doses of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy |
https://ClinicalTrials.gov/show/NCT01703988 |
Completed |
Biogen |
2015-01-31 |
| NCT01645787 |
Short and Long Term Treatment With 4-AP in Ambulatory SMA Patients |
https://ClinicalTrials.gov/show/NCT01645787 |
Completed |
Columbia University |
2015-09-30 |
| NCT01611610 |
Clinical Assessment of Spinal Muscular Atrophy Type II and III (SMA Europe) |
https://ClinicalTrials.gov/show/NCT01611610 |
Completed |
Institut de Myologie, France |
2013-05-31 |
| NCT01522079 |
Spinal Muscular Atrophy and Cardiac Autonomic Function |
https://ClinicalTrials.gov/show/NCT01522079 |
Completed |
Centro Universitário Augusto Motta |
2011-08-31 |
| NCT01494701 |
An Open-label Safety, Tolerability, and Dose-range Finding Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA) |
https://ClinicalTrials.gov/show/NCT01494701 |
Completed |
Biogen |
2013-01-31 |
| NCT04115475 |
New Imaging Biomarkers for Muscular Diseases - Multispectral Optoacoustic Imaging in Spinal Muscular Atrophy |
https://ClinicalTrials.gov/show/NCT04115475 |
Active, not recruiting |
University of Erlangen-Nürnberg Medical School |
2020-01-30 |
| NCT04050852 |
Pulmonary Function Test Changes and Respiratory Muscle Strength Trends in Spinal Muscular Atrophy Patients Receiving Nusinersen Treatments |
https://ClinicalTrials.gov/show/NCT04050852 |
Recruiting |
NYU Langone Health |
2020-07-17 |
| NCT03988907 |
A Drug-drug Interaction Study With Risdiplam Multiple Dose and Midazolam in Healthy Participants |
https://ClinicalTrials.gov/show/NCT03988907 |
Completed |
Hoffmann-La Roche |
2019-09-29 |
| NCT03970135 |
Fat and Glucose Metabolism in Fed and Fasted State in Patients With Low Skeletal Muscle Mass |
https://ClinicalTrials.gov/show/NCT03970135 |
Completed |
Rigshospitalet, Denmark |
2017-11-30 |
| NCT03921528 |
An Active Treatment Study of SRK-015 in Patients With Type 2 or Type 3 Spinal Muscular Atrophy |
https://ClinicalTrials.gov/show/NCT03921528 |
Active, not recruiting |
Scholar Rock, Inc. |
2021-01-31 |
| NCT03920865 |
A Study to Investigate the Effect of Hepatic Impairment on the Pharmacokinetics and Safety and Tolerability of a Single Oral Dose of Risdiplam Compared to Matched Healthy Participants With Normal Hepatic Function |
https://ClinicalTrials.gov/show/NCT03920865 |
Completed |
Hoffmann-La Roche |
2020-01-02 |
| NCT03837184 |
Single-Dose Gene Replacement Therapy Using for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies |
https://ClinicalTrials.gov/show/NCT03837184 |
Recruiting |
AveXis, Inc. |
2021-04-01 |
| NCT03781479 |
Controlled Trial to Evaluate Amifampridine Phosphate in Spinal Muscular Atrophy Type 3 Patients |
https://ClinicalTrials.gov/show/NCT03781479 |
Recruiting |
Catalyst Pharmaceuticals, Inc. |
2020-05-15 |
| NCT03779334 |
A Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy |
https://ClinicalTrials.gov/show/NCT03779334 |
Recruiting |
Hoffmann-La Roche |
2021-06-21 |
| NCT03505099 |
Pre-Symptomatic Study of Intravenous Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy (SMA) for Patients With Multiple Copies of SMN2 |
https://ClinicalTrials.gov/show/NCT03505099 |
Active, not recruiting |
AveXis, Inc. |
2021-06-24 |
| NCT03461289 |
Single-Dose Gene Replacement Therapy Clinical Trial for Patients With Spinal Muscular Atrophy Type 1 |
https://ClinicalTrials.gov/show/NCT03461289 |
Active, not recruiting |
AveXis, Inc. |
2020-09-30 |
| NCT01233817 |
Progressive Strength Training in Spinal Muscular Atrophy |
https://ClinicalTrials.gov/show/NCT01233817 |
Completed |
University of Utah |
2013-06-30 |
| NCT01166022 |
Clinical Trial of Exercise in Patients With Spinal Muscular Atrophy (SMA) |
https://ClinicalTrials.gov/show/NCT01166022 |
Completed |
Columbia University |
2014-08-31 |
| NCT01028833 |
Effects of Power Mobility on Young Children With Severe Motor Impairments |
https://ClinicalTrials.gov/show/NCT01028833 |
Completed |
University of Oklahoma |
2012-12-31 |
| NCT02908685 |
A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy (SMA) Participants |
https://ClinicalTrials.gov/show/NCT02908685 |
Active, not recruiting |
Hoffmann-La Roche |
2019-09-06 |
| NCT02428673 |
Effects of Standing on Non-Ambulatory Children With Neuromuscular Conditions |
https://ClinicalTrials.gov/show/NCT02428673 |
Active, not recruiting |
Gillette Children’s Specialty Healthcare |
2019-03-31 |
| NCT04262570 |
Evaluation of Therapeutic Response in Spinal Muscular Atrophy Using Multispectral Optoacoustic Tomography (MSOT) and Magnetic Resonance Imaging (MRI) |
https://ClinicalTrials.gov/show/NCT04262570 |
Recruiting |
University of Erlangen-Nürnberg Medical School |
2021-12-31 |
| NCT00661453 |
CARNIVAL Type I: Valproic Acid and Carnitine in Infants With Spinal Muscular Atrophy (SMA) Type I |
https://ClinicalTrials.gov/show/NCT00661453 |
Completed |
University of Utah |
2012-05-31 |
| NCT00568802 |
A Pilot Therapeutic Trial Using Hydroxyurea in Type II and Type III Spinal Muscular Atrophy Patients |
https://ClinicalTrials.gov/show/NCT00568802 |
Completed |
Stanford University |
2010-03-31 |
| NCT00568698 |
A Pilot Therapeutic Trial Using Hydroxyurea in Type I Spinal Muscular Atrophy Patients |
https://ClinicalTrials.gov/show/NCT00568698 |
Completed |
Stanford University |
2012-02-29 |
| NCT00533221 |
Pilot Study of Growth Hormon to Treat SMA Typ II and III |
https://ClinicalTrials.gov/show/NCT00533221 |
Completed |
University Hospital Freiburg |
2010-02-28 |
| NCT00528268 |
Study to Evaluate Sodium Phenylbutyrate in Pre-symptomatic Infants With Spinal Muscular Atrophy |
https://ClinicalTrials.gov/show/NCT00528268 |
Completed |
University of Utah |
2013-12-31 |
| NCT00485511 |
A Trial of Hydroxyurea in Spinal Muscular Atrophy |
https://ClinicalTrials.gov/show/NCT00485511 |
Completed |
Kaohsiung Medical University Chung-Ho Memorial Hospital |
2009-06-30 |
| NCT00481013 |
Valproic Acid in Ambulant Adults With Spinal Muscular Atrophy |
https://ClinicalTrials.gov/show/NCT00481013 |
Completed |
University of Utah |
2009-12-31 |
| NCT00374075 |
Study of Safety and Dosing Effect on SMN Levels of Valproic Acid (VPA) in Patients With Spinal Muscular Atrophy |
https://ClinicalTrials.gov/show/NCT00374075 |
Completed |
University of Utah |
NA |
| NCT00324454 |
Levetiracetam for Cramps, Spasticity and Neuroprotection in Motor Neuron Disease |
https://ClinicalTrials.gov/show/NCT00324454 |
Completed |
Duke University |
2007-12-31 |
| NCT00227266 |
Valproic Acid and Carnitine in Patients With Spinal Muscular Atrophy |
https://ClinicalTrials.gov/show/NCT00227266 |
Completed |
University of Utah |
2007-11-30 |
| NCT00004771 |
Phase II Study of Leuprolide and Testosterone for Men With Kennedy’s Disease or Other Motor Neuron Disease |
https://ClinicalTrials.gov/show/NCT00004771 |
Completed |
Office of Rare Diseases (ORD) |
NA |